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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT02935400
Other study ID # 14-0001
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date April 28, 2014
Est. completion date December 31, 2025

Study information

Verified date April 2024
Source The University of Texas Medical Branch, Galveston
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

The long term objective of the research is to identify new biomarkers of disease activity in the human acute porphyrias. This pilot study is intended to provide pilot and feasibility data needed to plan larger and more definitive future studies.


Description:

This translational pilot research is based on preliminary results using animal models. The investigators will collect samples of blood, urine and feces from up to 50 participants with well documented acute porphyrias, at 2 expert sites that are members of the Porphyrias Consortium. Collection and analysis of these samples will be used to assess feasibility of performing such studies in humans with acute porphyrias, recognizing that these disorders are more heterogeneous than reproduced in animal models, and affect individuals who cannot all be studied simultaneously and in large groups. Therefore, we will assess the feasibility of methods for collecting, processing, storing and shipping samples at multiple study sites for later biomarker analysis. Larger and more definitive studies of biomarkers will be designed and implemented based on data and experience from this pilot-feasibility study.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 50
Est. completion date December 31, 2025
Est. primary completion date December 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: - Documented diagnosis of acute porphyria. 1. For AIP: Elevation in urine PBG, with normal or only slight increases in plasma and fecal porphyrins. Most (~90%) will have deficient activity of erythrocyte PBGD. 2. For HCP: Elevation in urine PBG, with substantial increases in fecal porphyrins (almost entirely coproporphyrin III). In the absence of skin photosensitivity, most will have normal or only slight increases in plasma porphyrins. 3. For VP: Elevation in PBG, with substantial increases in fecal porphyrins (mostly coproporphyrin III and protoporphyrin), increased plasma total porphyrins and a fluorescence emission maximum of diluted plasma at neutral pH near 626 nm. Exclusion Criteria: - Another medical condition that might confound the results, as judged by the investigator

Study Design


Intervention

Drug:
Hemin
Hemin will be administered under a separate protocol or for clinical treatment, and samples will be collected under this protocol before and after treatment.

Locations

Country Name City State
United States University of Texas Medical Branch Galveston Texas

Sponsors (1)

Lead Sponsor Collaborator
The University of Texas Medical Branch, Galveston

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Potential biomarkers Biomarkers Expression of heme biosynthetic and heat and stress response genes 10 days
See also
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Completed NCT02082860 - Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria Phase 1
Completed NCT03338816 - ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) Phase 3
Completed NCT02452372 - A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP) Phase 1
Completed NCT00418795 - Porphozym in the Treatment of Acute Attacks in AIP Phase 2/Phase 3
Active, not recruiting NCT01617642 - Dental Health, Diet, Inflammation and Biomarkers in Patients With Acute Intermittent Porphyria(AIP)
Completed NCT02076763 - Observational Study of Acute Intermittent Porphyria Patients N/A